Shares in Neuren Pharmaceuticals (ASX: NEU) were up 17% at the open after the biotech stock flagged productive discussions with the United States Food and Drug Administration (FDA) on a clinical trial linked to its drug candidate.
What appears to have excited investors this morning were revelations that the upcoming Phase 3 trial for NNZ-2591 - a potential treatment for Phelan-McDermid syndrome (PMS) - has been given the all clear from the FDA to proceed as planned.
Best estimates suggest that between 1 in 8,000 and 1 in 15,000 people have PMS, which has severe quality of life impacts on those living with it, including moderate to severe developmental and intellectual impairment and developmental delay.
Communication is one of the most impactful health concerns in PMS reported by caregivers.
Receptive communication, as measured by VABS-3 Receptive-Raw Score, is the ability to receive and understand non-verbal and verbal interactions which is a foundational skill for the development of learning, social interaction, and speech.
What makes Neuren's progress all the more significant and potentially commercially valuable is the absence of any current approved PMS treatments.
Building on strong Phase 2 results, the company's planned Phase 3 trial will assess changes in receptive communication and a global clinical impression of change, using tools already validated in the earlier study.
The company expects NNZ-2591 to qualify for orphan drug and rare paediatric disease designations.
Subject to a final FDA review the trial is expected to launch mid-year 2025.
Commenting on today’s announcement, Neuren CEO Jon Pilcher noted: “We are very pleased with the outcome of another constructive discussion with the FDA and are now excited to be able to move forward as planned with the first ever Phase 3 trial in children with Phelan-McDermid syndrome.”
Neuren’s financial strength means no additional funding is required to execute the program.
Neuren has a market cap of $1.3 billion, making it an ASX300 stock; the share price is down 48% in one year and down 14% year to date.
Late February saw Neuren announce receipt of cash in its first sales milestone payment, due for the first calendar year in which net sales of DAYBUETM (trofinetide) in North America exceed US$250 million.
Neuren’s partner Acadia Pharmaceuticals reports 2024 net sales of DAYBUE in the U.S. of US$348.4 million.
For the year ended 31 December 2024, Neuren reported total income of $227.8 million, $213.2 earned under the licence agreement with Acadia Pharmaceuticals.
Multiple indicators indicate a long-term bearish trend for the stock.
Consensus is Strong Buy.
This article does not constitute financial or product advice. You should consider independent advice before making financial decisions.
